Suchergebnisse - "Urea cycle disorders"

PubPharm (717)

1

Liver Disease in Urea Cycle Disorders : Noninvasive Biomarkers of Hepatic Fibrosis in Urea Cycle Disorders

enthalten in: ClinicalTrials.gov | 2024

2

Are asymptomatic carriers of OTC deficiency always asymptomatic? A multicentric retrospective study of risk using the UCDC longitudinal study database

enthalten in: Molecular genetics & genomic medicine | 2024

von Sen, K. | Izem, R. | Long, Y. | +31

3

The efficacy of Carbamylglutamate impacts the nutritional management of patients with N-Acetylglutamate synthase deficiency

enthalten in: Orphanet journal of rare diseases | 2024

von Singh, R. | Bourdages, M. | Kurtz, A. | +5

4

The efficacy of Carbamylglutamate impacts the nutritional management of patients with N-Acetylglutamate synthase deficiency

enthalten in: Orphanet journal of rare diseases | 2024

von Singh, R. | Bourdages, M. | Kurtz, A. | +5

5

An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency (OTC-HOPE) : A Phase I/II First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of a Single Intravenous (IV) Administration of ECUR-506 in Males Less Than 9 Months of Age With Genetically Confirmed Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency

enthalten in: ClinicalTrials.gov | 2024

6

Severity-adjusted evaluation of liver transplantation on health outcomes in urea cycle disorders

enthalten in: Genetics in medicine : official journal of the American College of Medical Genetics | 2024

von Posset, R. | Garbade, S. | Gleich, F. | +58

7

Impact of citrulline substitution on clinical outcome after liver transplantation in carbamoyl phosphate synthetase 1 and ornithine transcarbamylase deficiency

enthalten in: Journal of inherited metabolic disease | 2024

von Aldrian, D. | Waldner, B. | Vogel, G. | +20

8

Impact of supplementation with L-citrulline/arginine after liver transplantation in individuals with Urea Cycle Disorders

enthalten in: Molecular genetics and metabolism | 2024

von Posset, R. | Garbade, S. | Gleich, F. | +9

9

Mitochondrial targets in hyperammonemia : Addressing urea cycle function to improve drug therapies

enthalten in: Biochemical pharmacology | 2024

von Moedas, M. | Simões, R. | Silva, M.

10

HepaStem Long-Term Safety Registry : HepaStem Long-Term Safety Registry - Registry for Patients Who Have Been Administered HepaStem

enthalten in: ClinicalTrials.gov | 2024

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